. 2024 Dec 30;19:495. doi: 10.1186/s13023-024-03474-6

Table 4.

Study and treatment characteristics of the studies on the use of omaveloxolone by Friedreich’s Ataxia

Study	Inclusion criteria	Exclusion criteria	Treatment regimen	Randomization
Part 1 [10]	Genetically confirmed FA^a with an mFARS^b score 10–80 Age: ≥ 16 to ≤ 40 years Ability to complete maximal exercise testing, and able to ride an exercise ergometer (approximately 60 rpm) for 3 min with no additional resistance	History of clinically significant cardiac disease BNP^c level > 200 pg/mL Uncontrolled diabetes (HbA1c^d > 11.0%)	Cohorts (n = 8) at ascending dose levels for 12 weeks Two cohorts 160 mg once/day) Omav^e (n = 12) and PBO^f (n = 4), Two cohorts (300 mg once/day) Omav (n = 10) and PBO (n = 3)	3:1::Omav: PBO for each dose
Part 2 [23]	Confirmed disease for 3–5 years; mFARS score 10–80	Same as part 1 and individuals with severe foot deformity	150 mg capsule of Omav once daily	1:1::Omav: PBO Stratified as with pes cavus and without pes cavus
Part 3 [24]	Confirmed disease and completed MOXIe part 2 with follow up mFARS score 20–80	Same as part 2	150 mg capsule of Omav once daily	Not applicable

Study

Inclusion criteria

Exclusion criteria

Treatment regimen

Randomization

Part 1 [10]

Genetically confirmed FA^a with an mFARS^b score 10–80

Age: ≥ 16 to ≤ 40 years

Ability to complete maximal exercise testing, and able to ride an exercise ergometer (approximately 60 rpm) for 3 min with no additional resistance

History of clinically significant cardiac disease

BNP^c level > 200 pg/mL

Uncontrolled diabetes (HbA1c^d > 11.0%)

Cohorts (n = 8) at ascending dose levels for 12 weeks

Two cohorts 160 mg once/day) Omav^e (n = 12) and PBO^f (n = 4),

Two cohorts (300 mg once/day) Omav (n = 10) and PBO (n = 3)

3:1::Omav: PBO for each dose

Part 2 [23]

Confirmed disease for 3–5 years;

mFARS score 10–80

Same as part 1 and individuals with severe foot deformity

150 mg capsule of Omav once daily

1:1::Omav: PBO

Stratified as with pes cavus and without pes cavus

Part 3 [24]

Confirmed disease and completed MOXIe part 2 with follow up

mFARS score 20–80

Same as part 2

150 mg capsule of Omav once daily

Not applicable

^aFriedreich’s ataxia

^bmodified Friedreich’s Ataxia Rating Scale

^cB-type natriuretic peptide

^dHemoglobin A1C test

^eOmaveloxolone treatment

^fPlacebo